BridgeBio Pharma’s Origin Biosciences Presents New Data on the Natural History of Molybdenum Cofactor Deficiency (MoCD) Type A at the Society of the Study of Inborn Errors of Metabolism (SSIEM) Conference
The majority of patients had MoCD Type A (n=37), followed by MoCD Type B (n=16) and Other (n=12).
- The majority of patients had MoCD Type A (n=37), followed by MoCD Type B (n=16) and Other (n=12).
- Nearly all patients (n=57) had first presenting symptoms by Day 28 with median onset for MoCD Type A patients at 2days.
- 2Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency.
- Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study.